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Friday, January 18, 2008

Earlier this week, the US Supreme Court declined to hear the appeal of the Abigail Alliance--news from the LA Timeshere. The advocacy organization sought to change the Food and Drug Administration's policy of prohibiting the use of experimental and unapproved drugs to treat patients. While there are procedures on the books for compassionate use, which allows access for some patients to some drugs under some circumstances, the Abigail Alliance and its supporters believe that the current approach is too narrow, too onerous, and too time-consuming to help all those who could benefit. They sought to make access easier for terminally ill patients who are unable or ineligible to participate in clinical trials.

The Abigail Alliance makes a stirring argument: none of us would want to feel that we were unable to get access to the "silver bullet" that would make our loved one (or ourselves) well again or extend life because of some bureaucratic requirement. And certainly the story of Abigail Burroughs--by all accounts a lovely young woman, who died of a rare form of cancer when she was just 21--is terribly sad.

But does it mean FDA is wrong to deny access to unproven drugs? I don't think so. The FDA's mission is to ensure that the medicines and devices we use to improve human health are safe and effective. This is a vital function and one that must be preserved.

We--by which I mean the general public--have a tendency to assume that drugs are good, that they work, and that they generally don't make people sicker. This is not a great assumption: check out the news earlier this week about Zetia and Vytorin (cholesterol drugs that could actually increase atherosclerosis), or the Vioxx settlement (a pain-reliever that caused heart attacks and strokes in some patients), or reports that suggest that several common drugs for schizophrenia may increase diabetes risk, or fen-phen (an off-label use for weight loss caused permanent heart damage in some patients). These are just a few examples. Drugs can be dangerous. They can make people sicker, and in some cases, they can give people worse problems than they started out with.

"Aha!" the advocates say, "But what about if somebody is already dying? They're as sick as they're going to get. Surely they should be granted access!" Still, in general, I think not. Patients who are terminally ill are the very last people we should be treating with unproven agents. Clinical trials aren't all beer and skittles: If you doubt that drugs can be dangerous, check out reports of the TeGenero trials last summer, in which 6 previously healthy young men ended up in the ICU with permanent immune system damage. These were Phase I safety trials, not Phase II--the category the Alliance wants to make available--but my point is, should we really subject patients to such an ordeal in the pursuit of what is likely an understandable, but unrealizable, dream of a cure?

A key point to understand is that research is different from treatment. Research is directed toward the development of new solutions, and it may or may not benefit those individuals who volunteer to participate. It sounds harsh, but helping volunteers isn't the point of research. Treatment, on the other hand, is all about managing symptoms and effecting cures. It's just plain wrong to represent one as the other.

The legal questions are more subtle: argument in the original case focused on whether terminally ill patients have a Constitutional right to drugs that have passed Phase I clinical testing. Our very own Linda Glenn did a nice overview here.

5 comments:

*contemplate*I think I'm going to disagree, although I admit I don't fully back the Abigail Alliance. I'll also admit that my opinion is strongly shaped by having recently lost a loved one to a terminal illness.

Because of that experience, I'm firsthand aware of the impulse to run for the most promising cure, or even medication that will extend a good quality of life longer. Suddenly, going to Mexico in search of peach pit extract seems like a good idea - it's promising research, it's a gamble... but when everything else has tried and failed, you want to find something, anything.

And unfortunately that's a natural human impulse that's not going to go away, no matter what the Supreme Court rules, or the FDA decides. Legislating human behaviour rarely works. Sure, the patients might not be able to get access to the drug in question in the United States - but that doesn't mean they won't try to go elsewhere, or find something else.

Given this reality, it seems like the better idea is to set up some sort of guidelines (ones that are actually practical when applied to someone dying of a terminal illness, unlike the current compassionate use laws) that will allow people to act on that human impulse in as safe a manner as possible, to prevent people from being subject to exploitation by unscrupulous people who prey on the weak, and yes, if possible, contribute to the greater human good.

It's a problem that we're going to face more and more - how do you do a trial of a substance in humans, when the thing you're trying to cure is deadly? Eventually you have to test it on people with the disease - and some people will die. If you know you're going to die if nothing is done, and you're offered the chance to help others before you go - I think you'd find a lot of people accepting that offer, both on the off chance they might actually survive, but also on the idea of being able to leave a contribution greater than family and friends behind.

Ultimately, I think it's a twofold issue, in that allowing access to potentially beneficial medications means that people won't be rushing off to try the latest Mexican Spa medication (thus fulfilling a mandate of protecting the public), and secondly that it can contribute to the greater body of knowledge.

The needs of the many and the needs of the one do not always have to be at odds.

First, my sympathies to you for your loss, Kelly. And I do understand your position. I feel a lot of sympathy, actually, to the argument, but I still disagree. (Respectfully!)

To me, the idea of giving people access to unproven therapies--as therapies--is plainly a bad idea. I mean, on some level, how different *is* this from laetrile, etc.?

Additionally, I think it's important to note that such use is *not* research--and so it's not clear to me what we "learn" from those uses. The cases we're talking about are generally ones where the person has been deemed ineligible for the relevant clinical trial.

I just think there is a huge, and morally relevant difference, between what happens in research (and what research subjects can and should expect) and what happens in medical care.

Finally, the fact that someone wants something doesn't create an obligation on the part of society (or even drug companies or insurers) to provide it. My understanding is that this is where the legal case fell apart for the Alliance--they had to prove that patients had a positive right to these medications, and the court couldn't find one.

I worry, too, that the more we push FDA to open access, speed up approval processes, etc. the more we're going to end up with drugs in the market that end up hurting lots of people: the ones I listed in the original post are just the tip of the iceberg....there was another one in the news this a.m. about osteoporosis drugs.

The cases we're talking about are generally ones where the person has been deemed ineligible for the relevant clinical trial.Truth - but that's problematic in and of itself, the way people are chosen or deemed eligible for trials. There's been a lot written on just how uneven and poorly designed many of these trials and their exclusionary policies are. (Not to mention things like access to the areas doing the research trials - I'm not convinced someone should be excluded from a promising treatment because they don't live within 45 miles of a research facility.)

I think that the way the research system is set up right now certainly limits the data that can be of "hard use" - but I also think that hard research, especially that funded by the pharma companies, focuses entirely too much on padding the results to be as beneficial as possible (and that's what excludes many people from being brought into trials - they're "too sick" to make the numbers look good). Perhaps that's a bit on the conspiracist side, but...

I worry, too, that the more we push FDA to open access, speed up approval processes, etc. the more we're going to end up with drugs in the market that end up hurting lots of peopleI very much agree with this, actually - I think I'm just seeing it from the other side. I'd rather see approval for new drugs slowed down, compassionate use access made easier, more protection offered to companies that move slowly and show good research that's independently verified, and a host of other reforms to the system. I just think that one of those reforms needs to be a way to get experimental medication to those who do need it, for the reasons I listed above.

While I don't support all of the Abigail Alliance's desires, I did find that the need to prove a positive right to access of the medications a bit dodgy, just because as a general rule, we don't view medical care and treatment as a positive right in this country. So asking someone to prove a positive right in a system not predicated on such thought is kind of a losing battle to begin with.

Yeah, lots of good points here--in particular, your comment is dead on! Definitely a through-the-looking-glass quality to this whole debate.

I agree (it would be hard not to!) that research isn't working as well as it should and is designed largely for the convenience of the researchers. I'm just not sure that *less* regulation is the solution here.

At bottom, my concern is that we could end up in a situation where the drug companies are running wild--and getting to do so under the flag of "respect for patient autonomy." Very Orwellian, imo.

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